ataluren

PTC Therapeutics has begun submitting an application for U.S. approval of Translarna (ataluren) for Duchenne MD; completion of the application is slated for late 2015

posted on December 23, 2014 - 11:20am

New Jersey-based PTC Therapeutics has begun submitting a rolling new drug application (NDA) to the U.S. Food and Drug Administration (FDA) for the approval of its experimental drug for the treatment of Duchenne muscular dystrophy (DMD) caused by specific flaws (mutations) in the dystrophin gene.

The European Medicines Agency expressed a 'positive opinion' on conditional approval for ataluren (Translarna) to treat Duchenne MD; a decision is expected within three months

posted on May 23, 2014 - 3:24pm

The experimental Duchenne muscular dystrophy (DMD) drug ataluren (which has been given the brand name Translarna) has received encouragement from the European Medicines Agency (EMA) for conditional approval in European Union countries.

Ataluren (PTC124) for Duchenne-Becker MD related to premature stop codons did not receive approval in Europe prior to completion of an ongoing phase 3 trial

posted on January 31, 2014 - 10:15am

The experimental drug ataluren, in development for Duchenne muscular dystrophy (DMD) or Becker muscular dystrophy (BMD) caused by specific mutations in the gene for the dystrophin protein, will not receive conditional approval at this time from the European Medicines Agency (EMA). Ataluren's developer is requesting that the EMA re-examine the data.

An increase in dystrophin was seen in a foot muscle in 61 percent of boys with nonsense-mutation DMD treated with ataluren in an MDA-supported study

posted on January 15, 2014 - 9:47am

An increase in levels of the dystrophin protein was seen after treatment with the experimental drug ataluren (originally called PTC124) in an MDA-supported, phase 2a, open-label trial of 38 boys with Duchenne muscular dystrophy (DMD) caused by a specific type of mutation in the dystrophin gene.

Although the experimental Duchenne-Becker MD drug ataluren appeared to benefit walking ability in a clinical trial, some researchers question whether it works via stop codon read-through

posted on July 3, 2013 - 3:37pm

It's been widely accepted that the mechanism by which the experimental drug ataluren appears to benefit walking ability in Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD) is that it causes "read-through" of premature stop codons — genetic instructions that cause cells to stop making a protein before the process is complete.

PTC Therapeutics' large-scale multinational trial of ataluren for nonsense-mutation Duchenne or Becker MD has opened its first site in Cincinnati, Ohio

posted on May 8, 2013 - 10:31am

Update (Oct. 31, 2013): This story has been updated to reflect that several trial sites are open to new participants.
A large-scale, multinational, phase 3 trial of the experimental drug ataluren has opened at several sites.